Friday, December 09, 2011 5:59:45 PM
BMRN:
The BMRN R&D Day yesterday didn't have any earth shattering news but I found it quite interesting. I've gone over it for a second time now. Its on the long side 4 hours 10 minutes and is done as a video webcast. Without going into much detail here are some overall impressions:
1-Management really seems on top of GALNS to ensure success. Usually I am disappointed at slow enrollment but it appears they are deliberately going slow for a couple reasons. First getting a lot of site up and making sure each site executes properly (e.g. how tests are run, types of patients enrolled don't want ones who "fake" walk to qualify, etc.)
2-I was not too optimistic on PEG PAL potential and that certainly hasn't changed.
3-The PARP program seems more encouraging then I thought. I was starting to think the target was not as promising as initially thought because of some set backs by other molecules. However one of the presents talked a bit more on the mechanism and some Ovarian results that have me more optimistic.
4-It would seem BMRN is running to the challenge of finding more Orphan candidates. There latest one will involve CNS delivery certainly riskier than prior ERT's. The program is for Batten disease, Its actually sounds like a very terrible disease that is fatal in late childhood. The company played the video on the website http://noahshope.com/
5-I am cautiously optimistic on the Pompe Program but really no change from past few months.
Those interested can access the video webcast and PDF for the slides @
http://phx.corporate-ir.net/phoenix.zhtml?p=irol-eventDetails&c=106657&eventID=4249981
Now on to the Roche call
.
The BMRN R&D Day yesterday didn't have any earth shattering news but I found it quite interesting. I've gone over it for a second time now. Its on the long side 4 hours 10 minutes and is done as a video webcast. Without going into much detail here are some overall impressions:
1-Management really seems on top of GALNS to ensure success. Usually I am disappointed at slow enrollment but it appears they are deliberately going slow for a couple reasons. First getting a lot of site up and making sure each site executes properly (e.g. how tests are run, types of patients enrolled don't want ones who "fake" walk to qualify, etc.)
2-I was not too optimistic on PEG PAL potential and that certainly hasn't changed.
3-The PARP program seems more encouraging then I thought. I was starting to think the target was not as promising as initially thought because of some set backs by other molecules. However one of the presents talked a bit more on the mechanism and some Ovarian results that have me more optimistic.
4-It would seem BMRN is running to the challenge of finding more Orphan candidates. There latest one will involve CNS delivery certainly riskier than prior ERT's. The program is for Batten disease, Its actually sounds like a very terrible disease that is fatal in late childhood. The company played the video on the website http://noahshope.com/
5-I am cautiously optimistic on the Pompe Program but really no change from past few months.
Those interested can access the video webcast and PDF for the slides @
http://phx.corporate-ir.net/phoenix.zhtml?p=irol-eventDetails&c=106657&eventID=4249981
Now on to the Roche call
.
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